Krystal Biotech

Ultragenyx

This biopharmaceutical company develops and commercialises therapies for patients with serious rare and ultra-rare genetic diseases.

Castle Creek Biosciences

This company develops cell and gene therapies for patients suffering from rare genetic skin and connective tissue diseases.

Replay Therapeutics

This biotech company develops genome writing and delivery platforms to provide genetic medicines for patients with rare skin, eye and haematological diseases.

Mariposa Therapeutics

This biotechnology company develops a topical small-molecule therapy for patients with the rare genetic skin condition epidermolysis bullosa simplex.

Arcutis Biotherapeutics

This biopharmaceutical company develops and commercialises topical treatments for patients with chronic inflammatory skin diseases such as psoriasis, eczema, and seborrhoeic dermatitis.

Eton Pharmaceuticals

This company develops and commercialises prescription medicines to treat patients living with rare diseases.

Rocket Pharma

This company develops gene therapies for patients with rare and devastating genetic cardiovascular and haematological diseases.

Bluebird Bio

This company develops and manufactures gene therapies for patients with severe genetic diseases, including sickle cell disease and beta-thalassaemia.